The Rett Syndrome Global Registry

Status Recruiting

Clinical Trial Summary

The Rett Global Registry is a fully remote, global, caregiver-reported registry to collect information about caring for a loved one with Rett syndrome. In addition, caregivers have the ability to track and graph their loved one's symptoms and care strategies over time, store information for central access, and opt-in to complete medical record consolidation and summary. Qualified researchers and therapeutic developers may request access to de-identified aggregate information to further Rett research, or assist with clinical development planning to facilitate and expedite more effective clinical trials.

Clinical Trial Description

The Rett Syndrome Global Registry is a fully remote, global, caregiver-reported registry intended to meet the needs of caregivers, clinicians and researchers, and therapeutic developers with the goal to increase our understanding of this rare disorder, support better outcomes for those with Rett syndrome, and facilitate improved therapeutic development. The Rett Global Registry allows families to provide data about their experience with Rett syndrome to improve their loved one's care while contributing to research. Participants may opt in to track and graph symptoms and care strategies over time to support day-to-day care. Participants may also access aggregate data to see similarities and differences in care strategies and consolidate their personal information in a central location. Participants may opt-in to complete medical record consolidation and summary that is centrally accessible, able to be shared with care providers, and utilized for research. The registry database is designed and maintained to clinical trial standards and supports research and therapeutic development while meeting or exceeding federal privacy and confidentiality requirements. These datasets including caregiver-reported Rett syndrome progression, quality of life, at home day-to-day data, and consolidated medical records from office visits or hospital stays, provides unique and previously unused sources of information important for improving our understanding of Rett syndrome, allow additional avenues of research, and support therapeutic development. Specifically, the registry is intended to assist with clinical development planning, trial design, trial endpoints, and regulatory filings. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04900493
Study type	Observational [Patient Registry]
Source	Rett Syndrome Research Trust
Contact	Jana von Hehn, PhD
Phone	203-445-0041
Email	support@rettglobalregistry.org
Status	Recruiting
Phase
Start date	January 31, 2022
Completion date	June 30, 2031

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04988867` - An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome	Phase 2/Phase 3
Recruiting	`NCT00069550` - Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome	Phase 3
Enrolling by invitation	`NCT06139172` - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities	N/A
Not yet recruiting	`NCT04014985` - Patients With RETT Syndrome	N/A
Not yet recruiting	`NCT04041713` - A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome	Phase 2
Completed	`NCT02705677` - Biobanking of Rett Syndrome and Related Disorders
Terminated	`NCT02790034` - Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms	Phase 2/Phase 3
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Recruiting	`NCT05932589` - Neurophysiologic Biomarkers in Rett Syndrome
Recruiting	`NCT04463316` - GROWing Up With Rare GENEtic Syndromes
Completed	`NCT04776746` - Open-Label Extension Study of Trofinetide for Rett Syndrome	Phase 3
Completed	`NCT04181723` - Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)	Phase 3
Enrolling by invitation	`NCT03836300` - Parent and Infant Inter(X)Action Intervention (PIXI)	N/A
Completed	`NCT04514549` - ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
Completed	`NCT02738281` - Natural History of Rett Syndrome & Related Disorders
Terminated	`NCT02562820` - An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome	Phase 1
Completed	`NCT05687214` - Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome	N/A
Recruiting	`NCT06199700` - Esketamine for the Treatment of Rett Syndrome	Early Phase 1
Completed	`NCT03941444` - ANAVEX2-73 Study in Patients With Rett Syndrome	Phase 3
Recruiting	`NCT06346106` - The Diagnostic Experience of Male Rett Syndrome

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.